Notice of Intent to Publish a Funding Opportunity Announcement for Early-Stage Pharmacological Validation of Novel Targets and Accompanying Pre-Therapeutic Leads for Diseases of Interest to the NIDDK (R01)

The National Institute of Diabetes and Digestive and Kidney Disease intends to promote a new initiative by publishing a Funding Opportunity Announcement (FOA) to promote translation of basic science research into knowledge and tools that can be utilized to provide strong justification for later-phase drug discovery and development efforts in areas relevant to the National Institute of Diabetes and Digestive and Kidney Diseases. This includes obesity, diabetes and related aspects of endocrinology and metabolism, digestive diseases, liver diseases, nutrition, kidney and urological diseases, hematology, and specific aspects of cystic fibrosis. For additional information on disease areas of interest to the NIDDK, please see http://www2.niddk.nih.gov/Research/. Its objective is to stimulate research and technology development to promote the early-stage pharmacological validation of drug targets and accompanying small molecule chemical scaffolds or non-viral biologics that are not currently a focus within the biotechnology and pharmaceutical industries.

This Notice is being provided to allow potential applicants sufficient time to develop meaningful collaborations and responsive projects.

The FOA is expected to be published in October, 2012 with an expected receipt date in February, 2013.

This FOA will utilize the R01 activity code. Details of the planned FOA are provided below.

Research Initiative Details

Recent significant advances in genetics, the basic understanding of physiology, and the pathogenesis of disease coupled with technological advances in areas such as bioinformatics, chemical biology, synthetic chemistry, and protein engineering have provided a rich knowledge base and strong toolbox to identify and pursue new drug targets with the goal of generating new molecular therapies for the treatment of diseases. Despite the availability of these approaches, we are facing an increased failure rate of potential therapeutics for reasons of both safety and lack of human efficacy in clinical trials. Therefore, a critical need exists to develop better strategies to validate and prioritize targets and pre-therapeutic lead molecules based on their likelihood of success to safely alter disease progress and outcomes in humans. For information on disease areas of interest to the NIDDK, please see: http://www2.niddk.nih.gov/Research/.

This FOA is intended to extend the level of early-stage pharmacological validation of novel targets, novel chemical scaffolds, and novel non-viral biologics that are not the focus of significant efforts in the biotechnology and pharmaceutical industry for treatment of diseases within the mission of the NIDDK. Applications are expected to focus on early-stage pharmacological target validation which encompasses pre-clinical hypothesis testing to generate data that, over time, increases confidence that pharmacological manipulation of a target may be clinically efficacious and safe. This process occurs prior to clinical testing of a new compound but should include the use of human-derived data, tissues, cells, and systems. Applications should include a strong justification for the selection and appropriateness to the mission of the NIDDK of both the target and small molecule chemical scaffold or non-viral biologic as well as a discussion of the relevant prior art, intellectual property, and competitive landscape.

Examples of the level of early-stage target, chemical scaffold, or non-viral biologic validation intended to be pursued under this funding opportunity include, but are not limited to:
Elucidation of the initial structure-activity relationship and absorption, distribution, metabolism, excretion, and toxicity properties of a small molecule chemical scaffold to demonstrate its in vivo potential for further optimization;
Providing strong data, using well validated and predictive animal and/or human tissue-based models, that there is a high likelihood that a target's modulation by a small molecule or non-viral biologic will treat a disease.